Protein clumps in inclusion body and myofibrillar muscle disease
Protein aggregation in inclusion body and myofibrillar myopathies
Researchers are checking whether sticky protein clumps spread between muscle cells and cause damage in people with inclusion body myopathy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project ID | NIH-11305998 on NIH RePORTER |
What this research studies
Researchers will study muscle tissue taken from people with inclusion body myopathy (IBM) and related myofibrillar diseases to look for protein clumps such as TDP-43 and desmin. They will use lab experiments where patient-derived protein 'seeds' are introduced to healthy muscle cells or fibers to see if new clumps form. The team will test whether these seeds damage lysosomes and autophagy pathways and whether aggregates move from one muscle fiber to another. This approach combines patient biopsy samples with cell and tissue work to identify where therapies might block the spread of harmful protein aggregates.
Who could benefit from this research
Good fit: Adults diagnosed with inclusion body myopathy or related myofibrillar myopathies, especially those willing to provide a muscle biopsy sample, are the ideal candidates.
Not a fit: People without IBM or myofibrillar muscle diseases, or those unwilling to undergo biopsy, are unlikely to benefit directly from this work.
Why it matters
Potential benefit: If correct, stopping the spread of protein aggregates could slow or prevent muscle loss in people with IBM and point to new treatments.
How similar studies have performed: Related research has shown prion-like spread for some proteins in neurodegenerative diseases, but applying this mechanism to IBM muscle is relatively new and not yet proven.
Where this research is happening
Saint Louis, United States
- Washington University — Saint Louis, United States (Active)
Researchers
- Principal investigator: Weihl, Conrad C — Washington University
- Study coordinator: Weihl, Conrad C
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.